Generalised Lipodystrophy, Partial Lipodystrophy
Conditions
Keywords
Lipodystrophy, Metreleptin Registry
Brief summary
The study is a post-authorization, prospective, voluntary registry of patients treated with commercial metreleptin including, but not limited to, patients in the US and EEA.
Detailed description
This is a non-interventional, multicentre, prospective, observational study of patients receiving treatment with commercial metreleptin for lipodystrophy in the US (GL) and EEA (GL and PL). This registry will add to the knowledge about metreleptin gained from clinical trials by providing information on the incidence rates of acute pancreatitis associated with the discontinuation of metreleptin; and all cases of fatal or necrotizing pancreatitis, hepatic adverse events, hypoglycemia, hypersensitivity reactions, serious and severe infections, including serious infections resulting in hospitalization and death, loss of efficacy, new diagnoses of autoimmune disorders, exacerbation of existing autoimmune disorders, all cancers (excluding non-melanoma skin cancer) by cancer type, exposed pregnancies, and all-cause deaths, in patients treated with metreleptin in routine clinical practice.
Interventions
DRUGMetreleptin
Sponsors
Chiesi Farmaceutici S.p.A.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients treated with metreleptin through commercial supply at the time or before enrolment into registry * Patients who provide a written consent * Patient coming off metreleptin clinical studies and continuing or restarting treatment with metreleptin through commercial supply
Exclusion criteria
• Patients currently treated with an investigational agent as part of a clinical trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice | Adverse events will be collected from first dose to last visit - min. 10 years. For Prevalent Users: Serious adverse events will be collected from 6 months prior to enrolment to last visit - min. 10 years. | Estimation of the incidence rates of the following events of special interest in patients treated with metreleptin as part of current clinical practice: acute pancreatitis associated with discontinuation of metreleptin including fatal or necrotizing pancreatitis, hepatic adverse events, severe hypoglycemia, serious hypersensitivity reactions, serious and severe infections resulting in hospitalization or death, loss of efficacy, new diagnoses of autoimmune disorders, autoimmune disease exacerbation, all cancers, exposed pregnancies and pregnancy outcomes, all-cause deaths, medication errors. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Characteristics of the study population in terms of demographic profile, vital signs and clinical signs | Demographics and Vital Signs information will be collected at all study visits - min. 10 years | Description of the overall demographic and clinical characteristics in all patients treated with metreleptin (pattern of use analysis) |
| Characteristics of the study population in terms of clinical chemistry - Glycated Hemoglobin (HbA1c), Fasting Plasma Glucose (FPG) and Triglycerides (TG) | Clinical chemistry will be collected at all study visits - min. 10 years | Description of routine laboratory measurements that could be inferred as efficacy endpoints (including HbA1c, FPG, and TG) over time |
| Characteristics of the study population in terms of its use of metreleptin | Treatment information will be collected at all study visits - min. 10 years | Description of the metreleptin exposure information in all patients treated with metreleptin (pattern of use analysis) |
Countries
France, Germany, Italy, United Kingdom, United States
Contacts
CONTACTLori Hartnett
STUDY_DIRECTORLori Hartnett
Amryt Pharmaceuticals DAC
Outcome results
None listed